Amicus Therapeutics

Emerging Company in the Spotlight sponsored by JFK Communications.

BioNJ is pleased to shine the spotlight on one of our Innovation Members each month featuring their progress and work on behalf of Patients and their impact on the New Jersey life sciences ecosystem. This month’s Emerging Company in the Spotlight is Amicus Therapeutics. 

So many of our Members are making a dramatic impact on our State’s innovation ecosystem. We celebrate you! Amicus Therapeutics is one such company. 

Amicus Therapeutics

Amicus Therapeutics, Inc., a global biotechnology company at the forefront of developing advanced therapies to treat a range of devastating rare and orphan genetic diseases, prides itself on placing the needs of patients at the forefront of its efforts. A member of BioNJ for more than ten years, Amicus is headquartered in Cranbury, NJ, with locations in the UK, France, Germany, Italy, The Netherlands and Spain.

“New Jersey is a talent-rich environment, where access to technology and innovation enables companies like Amicus to push the boundaries of research and drug discovery,” says John F. Crowley, Chairman and CEO. “We are unique in that our scientists often work hand-in-hand with patient advisors when researching and discovering new approaches to complex diseases for people living with a variety of rare and difficult-to-treat conditions.

“The collective passion of our team fuels progress, which may enable us to deliver meaningful benefits in ways many other biotech companies have not been able to achieve,” adds Crowley.

Mr. Crowley became involved with biotechnology in 1998, when two of his children were diagnosed with Pompe disease, a severe and often fatal neuromuscular disorder. In his drive to find them a cure, Crowley left his position at Bristol-Myers Squibb and became an entrepreneur as the Co-founder, President and CEO of Novazyme Pharmaceuticals. The biotech start-up conducted research on an experimental enzyme replacement therapy (ERT) for Pompe disease, which Crowley credits as ultimately saving his children’s lives. After Novazyme was acquired by Genzyme Corporation in 2001, Crowley became senior vice president of Genzyme Therapeutics, where he continued to play a lead role in the development of a drug for Pompe disease.

Crowley and his family have been profiled on the front page of The Wall Street Journal and are the subjects of a book by Pulitzer Prize-winning journalist Geeta Anand, “The Cure: How a Father Raised $100 Million–and Bucked the Medical Establishment–in a Quest to Save His Children.” In 2010, a major motion picture, “Extraordinary Measures,” was inspired by the Crowley family’s journey: it starred Harrison Ford and Brendan Fraser.

Amicus Therapeutics’ lead product candidate, Galafold™ (migalastat), is a precision medicine that received full approval in the European Union and is in late-stage development in the U.S. and the rest of the world. Galafold™ will be used to treat adults with Fabry disease on the basis of their genetic mutation. Also in Amicus’ pipeline is SD-101, a product candidate in late-stage development that is a potential first-to-market therapy for the rare genetic connective tissue disorder epidermolysis bullosa (EB). The company is leveraging its biologics and Chaperone-Advanced Replacement Therapy (CHART™) platform technologies to develop novel ERT products for Fabry disease, Pompe disease, and other lysosomal storage disorders (LSDs). Amicus’ lead biologics program is ATB200/AT2221, a uniquely engineered ERT for Pompe disease that will be administered in combination with a pharmacological chaperone.

While leading the way in developing treatments for rare and orphan diseases, patient advocacy is the bedrock of Amicus Therapeutics’ corporate focus. The company collaborates with patient organizations, individual patients, their caregivers and healthcare practitioners to best understand and help support the rare disease community. “Our initiatives are designed to bring purpose and empowerment to help ease the difficulties experienced by individuals and families living with a rare disease,” notes Chief Patient Advocate Jayne C. Gershkowitz, who serves on the company’s Senior Leadership Team. “We are dedicated to being the bridge between patients, their families and resources, listening to their needs, and offering access to support and services across their disease experience. Across all company functions, our dedication to patients and their families is what drives us to truly provide healing beyond disease.”

Amicus Therapeutics established its Patient Advisory Board (PAB) Program in 2007 to gain insight from those affected by rare and orphan diseases, and to enhance the company’s partnership with and understanding of the patient community. PABs are disease-specific; their members represent a diversity of age, geography, and have a connection to the broader patient community beyond their own personal disease experience. Membership on an Amicus PAB is a two-year commitment and members are vetted through a nominating/application process. PAB members share their insights and knowledge on a range of topics including the design and implementation of clinical studies, patient advocacy activities, access and reimbursement issues and other topics.

For more information about Amicus Therapeutics go to www.amicusrx.com or to find out how you can help partner on issues in rare disease go to National Organization for Rare Disorders (NORD) or Global Genes® Allies in Rare Disease.